Back to resultsLong Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
Primary Purpose
Myasthenia Gravis, MuSK
Status
Active
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Amifampridine Phosphate
Sponsored by
About this trial
This is an interventional treatment trial for Myasthenia Gravis, MuSK
Eligibility Criteria
Inclusion Criteria:
- Participated in the MSK-002 study
Exclusion Criteria:
- Epilepsy and currently on medication
- Clinically significant abnormalities in ECG, in the opinion of the Investigator
Sites / Locations
- Cleveland Clinic
- Univerity of Pennsylvania
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
amifampridine phosphate
Arm Description
tablets equivalent to 10mg amifampridine, 3 to 4 times per day
Outcomes
Primary Outcome Measures
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Evaluate the long-term safety and tolerability amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. [ Time Frame: over 21 monts ] Descriptive statistics will be used to summarize study data.
Secondary Outcome Measures
Full Information
NCT ID
NCT03579966
First Posted
June 5, 2018
Last Updated
April 14, 2023
Sponsor
Catalyst Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03579966
Brief Title
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
Official Title
Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 1, 2018 (Actual)
Primary Completion Date
April 3, 2023 (Actual)
Study Completion Date
April 28, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Catalyst Pharmaceuticals, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Evaluate the long-term safety of amifampridine phosphate in the symptomatic relief of antibody positive MuSK-MG.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myasthenia Gravis, MuSK
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
70 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
amifampridine phosphate
Arm Type
Experimental
Arm Description
tablets equivalent to 10mg amifampridine, 3 to 4 times per day
Intervention Type
Drug
Intervention Name(s)
Amifampridine Phosphate
Intervention Description
tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Description
Evaluate the long-term safety and tolerability amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. [ Time Frame: over 21 monts ] Descriptive statistics will be used to summarize study data.
Time Frame
over 21 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Participated in the MSK-002 study
Exclusion Criteria:
Epilepsy and currently on medication
Clinically significant abnormalities in ECG, in the opinion of the Investigator
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Renato Mantegazza, MD
Organizational Affiliation
Carlo Besta Neurological Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Univerity of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
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